Base editing treats spinal muscular atrophy in mice
A one-time genome-editing treatment restores motor function and extends lifespan in an animal model of a neuromuscular disease that is the leading genetic cause of infant mortality.

Credit: Dan Wang and Ailing Du
Fluorescence image of the lumbar spinal cord in a mouse model of spinal muscular atrophy, showing cell nuclei (blue), astrocytes (red), and AAVs (green), viral packaging used to deliver the base-editing treatment.
Funding
This work was supported in part by the National Institutes of Health, the Bill and Melinda Gates Foundation, the Howard Hughes Medical Institute, and the Friedreich’s Ataxia Research Alliance (FARA).
Paper cited
Arbab M, Matuszek Z et al. Base editing rescue of spinal muscular atrophy in cells and in mice. Science. Online March 30, 2023. DOI:10.1126/science.adg6518